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HMA/VEN治疗方案调整及与 - 突变型急性髓系白血病相关的结果

HMA/VEN treatment modifications and associated outcomes in -mutant AML.

作者信息

Chin Kuo-Kai, Derkach Andriy, Famulare Christopher, Gupta Gaurav K, Borge P Dayand, Geyer Mark B, Goldberg Aaron D, Haque Tamanna, Park Jae H, Roeker Lindsey E, Tallman Martin S, Stahl Maximilian, Stein Eytan M

机构信息

Leukemia Service, Division of Hematologic Malignancies, Memorial Sloan Kettering Cancer Center, New York, NY, USA.

Department of Biostatistics and Epidemiology, Memorial Sloan Kettering Cancer Center, New York, NY, USA.

出版信息

Leuk Lymphoma. 2025 Feb;66(2):270-278. doi: 10.1080/10428194.2024.2411436. Epub 2024 Oct 14.

Abstract

Hypomethylating agents (HMA) and venetoclax (VEN) are commonly used in patients with -mutated (m) acute myeloid leukemia (AML) ineligible for induction chemotherapy. While prior studies demonstrated high response and survival rates with HMA/VEN in m AML, the impact of treatment modifications in real-world settings is unclear. We retrospectively reviewed 89 m AML patients treated with HMA/VEN from January 2018 to June 2023. CR/CRi rates were 76% in newly diagnosed (ND) and 55% in relapsed/refractory (R/R) patients, and median overall survival was 29.2 months (ND) and 17.1 months (R/R), respectively. Treatment modifications were common. Early VEN reductions were associated with lower response rates but not worse survival. Prolonged cycles were not associated with worse response rates or survival. Significant neutropenia and ED visits or unplanned hospitalizations were considerable before and after CR/CRi, though febrile neutropenia decreased afterward. HMA/VEN is efficacious, with treatment modifications not affecting survival, though long-term toxicities are notable.

摘要

低甲基化药物(HMA)和维奈克拉(VEN)常用于不符合诱导化疗条件的伴有突变(m)的急性髓系白血病(AML)患者。虽然先前的研究表明HMA/VEN治疗m AML有较高的缓解率和生存率,但在实际临床环境中治疗调整的影响尚不清楚。我们回顾性分析了2018年1月至2023年6月期间接受HMA/VEN治疗的89例m AML患者。新诊断(ND)患者的完全缓解(CR)/血细胞计数部分恢复的完全缓解(CRi)率为76%,复发/难治性(R/R)患者为55%,中位总生存期分别为29.2个月(ND)和17.1个月(R/R)。治疗调整很常见。早期减少VEN与较低的缓解率相关,但生存期并未更差。延长疗程与较差的缓解率或生存期无关。在CR/CRi前后,严重中性粒细胞减少以及急诊就诊或非计划住院情况较为常见,不过之后发热性中性粒细胞减少有所减少。HMA/VEN是有效的,治疗调整不影响生存期,尽管长期毒性较为显著。

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